# Hematologic Malignancies Program

> **NIH NIH P30** · UNIVERSITY OF PENNSYLVANIA · 2024 · $83,258

## Abstract

PROJECT SUMMARY – Hematologic Malignancies Program
The Hematologic Malignancies (HM) Program drives basic scientific discoveries and translates them into novel
therapeutics for patients with myeloid and lymphoid neoplasms. Formed in 1994, the Program has two
scientific aims: 1) Develop a mechanisms-based understanding of the genetic, cellular, and biochemical
processes regulating malignant hematopoiesis; and 2) Translate basic scientific discoveries into more effective
and less toxic therapies for hematologic malignancies. Areas of national expertise include molecular processes
(epigenetic, transcriptional, translational, signaling) underlying malignant hematopoiesis; hematopoietic stem
cell biology and transplantation; molecular therapeutics; and immune-based therapies. Annual average accrual
to interventional clinical trials has more than doubled this funding period compared to the prior, with heavy
emphasis on investigator-initiated trials. Program members are heavy users of Shared Resources (e.g.
Transgenic and Chimeric Mouse, Flow Cytometry and Cell Sorting, Human Immunology, Biostatistics and
Bioinformatics, and Genomic Analysis) and have wide-ranging collaborations with members of multiple other
Programs. The Program is co-led by Dr. Edward Stadtmauer, an expert in clinical trials of novel therapeutics
for hematologic malignancies and blood and marrow transplantation, and Dr. Nancy Speck, an internationally
known basic scientist working in the area of hematopoietic stem cells and leukemia. The Program's 29
members come from seven departments in the Perelman School of Medicine. Investments by the ACC include
an innovative Hematologic Malignancies Translational Center of Excellence (TCE) co-led by Dr. Stadtmauer,
as well as an emerging Lymphoid Malignancy TCE co-led by Drs. Stephen Schuster and Megan Lim. These
and other investments have galvanized an already successful Program by expanding resources for laboratory,
tissue banking, and clinical research and providing pilot grant funding. Scientific accomplishments this funding
period include studies garnering multiple FDA approvals (CAR T cell therapy for adults with relapsed and
refractory B cell non-Hodgkin's lymphoma; gilteritinib for refractory acute myeloid leukemia with FLT3
mutations; and selinexor, a first-in-class oral nuclear transport inhibitor for `penta-refractory' multiple myeloma).
Basic discoveries revealed novel tumor-cell intrinsic pathways such as the demonstration that kelch-like 6
(KLHL6) functions as an E3 ubiquitin ligase, and KLHL6 mutations in lymphoma increase NF-κB signaling.
Weekly seminars, collaborative grants, weekly clinical working group meetings, and an annual research retreat
facilitate member interactions. Currently, members have research funding totaling $16.2M (direct), of which
$5.8M is peer-reviewed and $1.9M is from the NCI. There are 20 R01-equivalents held by our members.
During the project period, members published 450 cancer relevant publications,...

## Key facts

- **NIH application ID:** 10772995
- **Project number:** 5P30CA016520-48
- **Recipient organization:** UNIVERSITY OF PENNSYLVANIA
- **Principal Investigator:** Edward A Stadtmauer
- **Activity code:** P30 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $83,258
- **Award type:** 5
- **Project period:** 1997-01-15 → 2025-11-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10772995

## Citation

> US National Institutes of Health, RePORTER application 10772995, Hematologic Malignancies Program (5P30CA016520-48). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10772995. Licensed CC0.

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